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Researchers discover test to predict which patients with rare blood disease will respond to only FDA-approved treatment, and identify alternative therapy

New research has uncovered a precision medicine test using blood proteins to identify a novel patient subgroup of idiopathic multicentric Castleman disease (iMCD), a rare blood disorder, who are more likely to respond to siltuximab, the only FDA approved treatment for the disease. Prior research suggests that half of patients do not respond to the monoclonal antibody treatment, siltuximab. For those patients, rapid administration of other treatments is needed to prevent deterioration, so understanding who is likely to benefit is critical. This study also revealed that an existing drug approach, Janus kinase (JAK) inhibitors, which are already approved for treating certain cancers and rheumatoid arthritis, are a promising alternative treatment option for patients who do not respond to siltuximab. The study, is the largest to date for iMCD.

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